Welcome and thank you for reaching out to Cure SMA!

The diagnosis of spinal muscular atrophy (SMA) can be confusing, scary, frustrating, pretty much the whole gamut of emotions, but you are not alone. You are now part of Cure SMA – A network of individuals with SMA, families, researchers, clinicians, and other health professionals who are determined to make a difference.

Founded by a group of parents in 1984, Cure SMA will provide unbiased support for all those dealing with SMA and will fund the most promising research. We have included many of our resources for you to use and share below. Please do not hesitate to contact us with any questions.

Research & Treatments

To date, Cure SMA has invested more than $85 million in research (click here to see our current and recent funding). The basic research that Cure SMA has funded, through research grants to institutions around the world, has delivered major discoveries:

  • We know the cause of SMA, which means we can develop treatments that correct the underlying cause of the disease rather than just reduce symptoms.
  • We identified a “back-up gene” for SMA, which means we have a straightforward drug target already in the body: a built-in switch for new therapies to work on.

Using this knowledge, we now have multiple therapies approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of SMA, over half a dozen clinical trials testing SMA therapies, and more than 15 total programs in various stages of drug development. Click here to learn more about the drug pipeline.

Our research approach funds programs at early stages, and then partners with companies to take them through clinical trials. Even with our current progress in adding new programs and advancing therapies, we believe it is critical to do more. We are not slowing down, and we will continue to build upon our current momentum.

Support Resources

Cure SMA, At a Glance

Click here to access and download all of our Cure SMA Care Series booklets in a variety of languages. These discuss topics from genetics of SMA, breathing basics, musculoskeletal system, and much more!

Cure SMA is committed to addressing the needs of individuals and families affected by SMA. To help reach this goal, we have developed a Newly Diagnosed Survey. By sharing your information here, you will help the scientific and research communities and accelerate therapy development for SMA.

To learn more about supporting Cure SMA’s fundraising efforts, click here.

Connect with Cure SMA on social media!

Cure SMA also has created a mobile app for the SMA community. The Cure SMA Guide app is a family support program that takes a new approach to provide useful tools and information related to SMA care, to use at home and on-the-go. Please download our app directly from the Apple Store or Google Play Store for Android devices. You can also visit this link to view the Cure SMA Guide on any compatible web browser.

Annual SMA Conference

All individuals with SMA, families, and caregivers are invited to Cure SMA’s Annual SMA Conference! If you have any questions about the conference, please email [email protected].


This is Cure SMA’s publication for individuals with SMA and families.

Click here to view all of our editions of Directions.


This is Cure SMA’s publication that shares research updates.

Click here to view all of our editions of Compass.

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